FDA de­rails Acor­da's mar­ket­ing pitch, cre­at­ing a cri­sis as flag­ship drug founders

Acor­da just en­tered a world of hurt.

With its patent pro­tec­tion crum­bling around its main­stay drug, the FDA has re­fused to file its ap­pli­ca­tion for CVT-301, the res­cue pro­gram that was des­ig­nat­ed to take the lead drug’s place. The RTF cre­ates an em­bar­rass­ing and per­haps crip­pling prob­lem for the biotech, which is fight­ing a last-ditch bat­tle to main­tain patent pro­tec­tion on its one big drug, Ampyra.

Ron Co­hen

Ac­cord­ing to Acor­da, the FDA says the com­pa­ny’s mar­ket­ing ap­pli­ca­tion, rushed to reg­u­la­tors as the com­pa­ny re­struc­tured and down­sized, is in­com­plete. Reg­u­la­tors said they were un­able to do a prop­er re­view as they don’t know what date Acor­da’s man­u­fac­tur­ing cen­ter will be ready for in­spec­tion and have a “ques­tion re­gard­ing the sub­mis­sion of the drug mas­ter pro­duc­tion record.”

Acor­da re­port­ed that the FDA al­so re­quest­ed ad­di­tion­al in­for­ma­tion at re­sub­mis­sion, which was not part of the ba­sis for the RTF.

Its shares quick­ly plunged 26% on the news.

Nor­mal­ly, a de­lay like this would be seen as a sticky prob­lem, but one that could po­ten­tial­ly be re­solved with­out huge de­lays. The prob­lem for Acor­da, though, is that the com­pa­ny has vir­tu­al­ly no lee­way for a sna­fu like this. Not on­ly does it leave Acor­da’s ex­ec­u­tive team with a black eye on ex­e­cu­tion, it cre­ates se­ri­ous doubts about its abil­i­ty to quick­ly and ef­fi­cient­ly line up a re­place­ment drug for its sales team by next sum­mer, as the lit­i­ga­tion over its patents for Ampyra comes to a head.

Leerink’s Paul Mat­teis found the whole thing puz­zling and dis­con­cert­ing, con­sid­er­ing the like­li­hood of a loom­ing ap­proval. And he still has ques­tions about what is go­ing on at Acor­da.

It seems like the best case sce­nario would be a re-sub­mis­sion around the end of this year, and its en­cour­ag­ing that FDA did not make any re­quests re­gard­ing the ef­fi­ca­cy or safe­ty da­ta. Nonethe­less, even af­ter speak­ing with the com­pa­ny, the RTF is mys­te­ri­ous to us, and we ex­pect the stock to be un­der pres­sure un­til opac­i­ty is lift­ed.

As I re­port­ed in June, while re­view­ing the late-stage da­ta sub­mit­ted to the FDA on the in­haled lev­odopa, any set­back on CVT-301 would be a cat­a­stro­phe for Acor­da.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

Now Acor­da is left to des­per­ate­ly seek a quick meet­ing with reg­u­la­tors to clear up any of these ques­tions. But the FDA is not known for scram­bling a re­sponse in any of these sit­u­a­tions, leav­ing Acor­da’s time­line in the air just when it most needs cer­tain­ty. Just weeks ago one of its biggest in­vestors pushed CEO Ron Co­hen to auc­tion the com­pa­ny off rather than risk the con­se­quences of fail­ing at its high wire act. The com­pa­ny pushed back, but a sale may come back on the ta­ble now whether or not it likes the idea.

“We will work with the FDA as quick­ly as pos­si­ble to ad­dress the open is­sues and to clar­i­fy the path to suc­cess­ful­ly re-file our ap­pli­ca­tion,” said Co­hen in a state­ment. “We re­main con­fi­dent in IN­BRI­JA’s da­ta pack­age and its promise as an im­por­tant new ther­a­py for peo­ple with Parkin­son’s dis­ease. We see tremen­dous long-term val­ue in its sol­id clin­i­cal pro­file, sig­nif­i­cant com­mer­cial op­por­tu­ni­ty and strong IP, and we re­main fo­cused on work­ing to bring pa­tients this im­por­tant new ther­a­py.”

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Top an­a­lyst of­fers a rare, up­beat in­ter­pre­ta­tion of Ab­b­Vie’s $63B Al­ler­gan deal — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took at 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Image: Chris Varma. Frontier

UP­DAT­ED: Chris Var­ma un­veils MP­M's lat­est start­up — eye­ing 'un­drug­gable' can­cer tar­gets and pow­ered by ma­chine learn­ing, $67M

Two years af­ter MPM Cap­i­tal en­list­ed Chris Var­ma on its busy on­col­o­gy team, the for­mer en­tre­pre­neur-in-res­i­dence is un­veil­ing his first ven­ture project out of his new stomp­ing grounds in the Bay Area: Fron­tier Med­i­cines.

For Var­ma, who’s al­so co-found­ed Blue­print Med­i­cines and built com­pa­nies at Third Rock and Flag­ship, this marks an­oth­er op­por­tu­ni­ty to ap­ply some cut­ting-edge sci­ence to “sev­er­al of the most im­por­tant and dif­fi­cult tar­gets in can­cer” — tar­gets that oth­ers have tried to tack­le with more clas­si­cal meth­ods and failed. The launch round comes in at $67 mil­lion, which should go some way in scaf­fold­ing a pre­clin­i­cal pipeline and push one or more as­sets in­to the clin­ic three years from now, he tells me.

The top 15 mega-deals in bio­phar­ma: Ab­b­Vie and Bris­tol-My­ers ac­qui­si­tions stir fresh de­bate over what's too big to buy

The debate over what’s too big to buy in biotech is back. A number of top analysts went right after AbbVie’s rationale for the Allergan deal today, just as Bristol-Myers Squibb stirred immediate debate over the worth and wisdom of acquiring Celgene.

To help provide some added context to this discussion, we asked DealForma chief Chris Dokomajilar to look over the past decade of major M&A in biopharma to decipher the top 15 plays.

The new numbers, unadjusted for inflation, harken back to the days of the Pfizer-Wyeth buyout and Merck’s decision to absorb Schering-Plough — both triggered in 2009. The heat over those acquisitions made the big pharma mega-deal highly unpopular for most everyone — except Pfizer — as industry leaders swore off almost all but the handy bolt-on acquisition.

Until recently.

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